GEN News Highlights

Geron to Develop Human ESC-Derived Chondrocytes for Cartilage Repair

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Geron has inked deals to develop human embryonic stem cell-derived chondrocytes for the treatment of cartilage damage and joint disease. The firm entered into a worldwide, exclusive license agreement with the University of Edinburgh covering technology that allows the efficient production of chondrocytes from human embryonic stem cells (ESCs).

The technology was developed in the laboratory of professor Brendon Noble, as part of a research collaboration between Geron and the university. The research was also supported by the UK Stem Cell Foundation, with funding from Scottish Enterprise and the Medical Research Council.

The agreement with the University of Edinburgh gives Geron the right to develop therapeutic applications of the technology. Additionally, it is expected to enhance the company’s program to develop cell-replacement therapies for orthopedic indications such as osteoarthritis.

Geron also established a development collaboration with the University Campus Suffolk (UCS), where professor Noble currently works. The project will be based within the new biotechnology unit at UCS, which has been supported by Suffolk County Council and the East of England Development Agency. UCS will also provide equipment for research. The program will be jointly funded by Geron and UCS, with the firm financing direct costs of development work at UCS.

Preclinical studies conducted by Professor Noble’s group have shown that injection of hESC-derived chondrocytes into damaged cartilage of the knee joint of immunocompetent rats produced well-integrated cartilage. Full repair of the lesion for at least nine months was demonstrated.

“The research in the U.K. has demonstrated that hESC-derived chondrocytes can generate cartilage that appears virtually indistinguishable from host tissue within months after implantation in damaged tissue in a rodent model,” reports Jane S. Lebkowski, Geron’s senior vp and CSO, regenerative medicine. “Current studies are being conducted in a large animal model to assess cartilage repair and function in a model resembling cartilage damage in the human knee. The next phase of the program will involve development of scaled manufacturing of the chondrocytes to support preclinical studies and eventual clinical testing.”

Geron is developing ESC-based therapeutics for cancer and chronic degenerative diseases including spinal cord injury, heart failure, and diabetes. Its lead candidate, GRNOPC1, recently gained approval from the FDA to move into Phase I testing, making the company the first to conduct clinical investigations of an ESC treatment.

GRNOPC1, an hESC-derived oligodendrocyte progenitor cell therapy, is being evaluated in patients with scale grade A subacute thoracic spinal cord injuries. Secondary endpoints will assess efficacy of the treatment in terms of improved neuromuscular control or sensation in the trunk or lower extremities.

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